BAX 326 Pediatric Study
Sponsor
Baxalta now part of Shire (Industry)
Overall Status
Completed
CT.gov ID
NCT01488994
Collaborator
(none)
23
11
2
16.8
2.1
0.1
Study Details
Study Description
Brief Summary
The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2/Phase 3 |
Detailed Description
The secondary outcome measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours (h) Post-infusion analysis was not done due to the different time-points for the last PK blood sample, AUC0-72 h was redundant and only total AUC was included in the PK analysis.
Study Design
Study Type:
Interventional
Actual Enrollment
:
23 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
BAX 326 (Recombinant Factor IX): A Phase 2/3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity in Previously Treated Pediatric Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level 1-2%) Hemophilia B
Actual Study Start Date
:
Dec 20, 2011
Actual Primary Completion Date
:
May 14, 2013
Actual Study Completion Date
:
May 14, 2013
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: BAX326 < 6 years of age
|
Biological: BAX326
All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.
Other Names:
|
| Experimental: BAX326 6 to <12 years of age
|
Biological: BAX326
All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Adverse Events (AEs) Possibly or Probably Related to BAX326 [Throughout study period (approximately 17 months)]
Secondary Outcome Measures
- Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose) [Within 30 mins pre-infusion and 4 post-infusion timepoints]
- Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose) [Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.]
- Pharmacokinetics (PK): Mean Residence Time (MRT) [Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.]
Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC)
- Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL) [Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.]
Computed as the dose divided by total Area under the curve (AUC)
- Pharmacokinetics (PK): Incremental Recovery (IR) [Within 30 mins pre-infusion and 30 mins post-infusion]
The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose
- Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2) [Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.]
Calculated as log_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model
- Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) [Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.]
Computed as Clearance (CL) * Mean residence time (MRT)
- Pharmacokinetics (PK): Incremental Recovery (IR) Over Time [Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26.]
IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants > 6 years of age; pediatric participants 6 to <12 years of age; pharmacokinetic Full Analysis Set (PKFAS).
- Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode [Throughout study period (approximately 17 months)]
- Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed [Throughout study period (approximately 17 months)]
Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring. - Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. - Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens.
- Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR) [Throughout study period (approximately 17 months)]
The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) * 365.25.
- Consumption of BAX326: Number of Infusions Per Month [Throughout study period (approximately 17 months)]
- Consumption of BAX326: Number of Infusions Per Year [Throughout study period (approximately 17 months)]
- Consumption of BAX326: Weight-adjusted Consumption Per Month [Throughout study period (approximately 17 months)]
- Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized) [Throughout study period (approximately 17 months)]
- Consumption of BAX326: Weight-adjusted Consumption Per Event [Throughout study period (approximately 17 months)]
Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs).
- Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) [Throughout study period (approximately 17 months)]
- Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX) [Throughout study period (approximately 17 months)]
If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data.
- Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis [Throughout study period (approximately 17 months)]
- Safety: Number of Participants With Thrombotic Events [Throughout study period (approximately 17 months)]
- Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs [Throughout study period (approximately 17 months)]
Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters
- Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) [Throughout study period (approximately 17 months)]
If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay.
- Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score [Baseline and 6 months]
For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline.
- Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score [Baseline and 6 months]
The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.
- Health Resource Use: Number of Hospitalizations [Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26]
The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.
- Health Resource Use: Length of Hospitalization [Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26]
The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.
- Health Resource Use: Unscheduled Doctor's Office Visits [Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26]
The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.
- Health Resource Use: Emergency Room Visits [Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26]
The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.
- Health Resource Use: Days Lost From School [Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26]
The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set.
Eligibility Criteria
Criteria
Ages Eligible for Study:
N/A
to 12 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Main Inclusion Criteria:
-
Participant and/or legal representative has/have voluntarily provided signed informed consent
-
Participant has severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B
-
Participant is < 12 years old at the time of screening
-
Participant has no evidence of a history of FIX inhibitors (based on the participant's medical records)
-
Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm^3
Main Exclusion Criteria:
-
Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 Bethesda Unit (BU)
-
Participant has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
-
Participant has evidence of an ongoing or recent thrombotic disease
-
Participant has an inherited or acquired hemostatic defect other than hemophilia B
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | LNJP Maulana Azad Medical College & Associated Hospitals | New Delhi | India | 110002 | |
| 2 | University Pediatric Hospital | Krakow | Poland | 30-663 | |
| 3 | Stanislaw Popowski Provincial Specialist Pediatric Hospital | Olsztyn | Poland | 10-561 | |
| 4 | Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin | Szczecin | Poland | 71-252 | |
| 5 | S.C. Sanador SRL | Bucharest | Romania | 11156 | |
| 6 | Louis Turcanu Emergency Children's Hospital | Timisoara | Romania | 300011 | |
| 7 | Regional Clinical Hospital | Ekaterinburg | Russian Federation | 620149 | |
| 8 | Pediatric Regional Clinical Hospital, Hematology Department | Krasnodar | Russian Federation | 350007 | |
| 9 | Republican Center for Hemophilia Treatment | St. Petersburg | Russian Federation | 195213 | |
| 10 | State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine" | Lviv | Ukraine | 79044 | |
| 11 | Manchester Children´s Hospital | Manchester | United Kingdom | M13 9WL |
Sponsors and Collaborators
- Baxalta now part of Shire
Investigators
- Study Director: Study Director, Takeda
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Baxalta now part of Shire
ClinicalTrials.gov Identifier:
NCT01488994
Other Study ID Numbers:
- 251101
- 2011-002437-19
First Posted:
Dec 9, 2011
Last Update Posted:
May 20, 2021
Last Verified:
Apr 1, 2021
Study Results
Participant Flow
| Recruitment Details | Enrollment was conducted at 11 clinical sites in 6 countries (United Kingdom, Poland, Romania, Russian Federation, Ukraine, India). A total of 23 participants were enrolled in the study. Of these, 11 were < 6 years of age and 12 were 6 to <12 years of age. |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to <12 Years of Age |
|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. |
| Period Title: Overall Study | ||
| STARTED | 11 | 12 |
| COMPLETED | 11 | 11 |
| NOT COMPLETED | 0 | 1 |
Baseline Characteristics
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to <12 Years of Age | Total |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to <12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Total of all reporting groups |
| Overall Participants | 11 | 12 | 23 |
| Age (years) [Mean (Full Range) ] | |||
| Mean (Full Range) [years] |
3.83
|
9.8
|
6.94
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
0
0%
|
0
0%
|
0
0%
|
| Male |
11
100%
|
12
100%
|
23
100%
|
Outcome Measures
| Title | Adverse Events (AEs) Possibly or Probably Related to BAX326 |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full analysis set |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number [AEs considered related to BAX326] |
0
|
0
|
0
|
| Title | Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose) |
|---|---|
| Description | |
| Time Frame | Within 30 mins pre-infusion and 4 post-infusion timepoints |
Outcome Measure Data
| Analysis Population Description |
|---|
| On completion of the study, prospective changes to the planned statistical analysis were made not to analyze AUC 0-72h due to the different time points for the last PK blood sample. Only total AUC [i.e. AUC 0-infinity] was included in the PK analysis. |
| Arm/Group Title | Overall Study Arm |
|---|---|
| Arm/Group Description | No participants |
| Measure Participants | 0 |
| Title | Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose) |
|---|---|
| Description | |
| Time Frame | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [IU*hour (hr)/dL] |
723.7
(119)
|
886
(133.66)
|
808.4
(149.14)
|
| Title | Pharmacokinetics (PK): Mean Residence Time (MRT) |
|---|---|
| Description | Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC) |
| Time Frame | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [hours (hr)] |
30.62
(3.266)
|
25.31
(1.830)
|
27.85
(3.726)
|
| Title | Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL) |
|---|---|
| Description | Computed as the dose divided by total Area under the curve (AUC) |
| Time Frame | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [dL/(kg*hr)] |
0.1058
(0.01650)
|
0.0874
(0.01213)
|
0.0962
(0.01689)
|
| Title | Pharmacokinetics (PK): Incremental Recovery (IR) |
|---|---|
| Description | The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose |
| Time Frame | Within 30 mins pre-infusion and 30 mins post-infusion |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | BAX326 < 6 Years of Age | BAX326 6 to <12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 10 | 12 | 23 |
| Mean (Standard Deviation) [IU/dL : IU/kg] |
0.586
(0.1320)
|
0.731
(0.1615)
|
0.665
(0.1632)
|
| Title | Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2) |
|---|---|
| Description | Calculated as log_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model |
| Time Frame | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [hours (hr)] |
27.67
(2.658)
|
23.15
(1.582)
|
25.31
(3.130)
|
| Title | Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) |
|---|---|
| Description | Computed as Clearance (CL) * Mean residence time (MRT) |
| Time Frame | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [dL/kg] |
3.225
(0.5233)
|
2.209
(0.3165)
|
2.695
(0.6662)
|
| Title | Pharmacokinetics (PK): Incremental Recovery (IR) Over Time |
|---|---|
| Description | IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants > 6 years of age; pediatric participants 6 to <12 years of age; pharmacokinetic Full Analysis Set (PKFAS). |
| Time Frame | Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26. |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Pharmacokinetic Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who had at least one plasma factor IX activity level available during post-infusion timepoints after infusion of study product. |
| Measure Participants | 11 | 12 | 23 |
| Baseline (N=10, 12, 22) |
0.586
(0.1320)
|
0.731
(0.1615)
|
0.665
(0.1632)
|
| Week 5 (N=11, 12, 23) |
0.630
(0.1028)
|
0.726
(0.1291)
|
0.680
(0.1245)
|
| Week 13 (N=10, 11, 21) |
0.676
(0.1211)
|
0.733
(0.1400)
|
0.706
(0.1313)
|
| Week 26 (N=10, 11, 21) |
0.647
(0.1274)
|
0.795
(0.1445)
|
0.724
(0.1533)
|
| Title | Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants in the Full Analysis Set who had bleeding episodes |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 7 | 7 | 14 |
| Bleeding Episodes controlled with 1 infusion |
9
|
6
|
15
|
| Bleeding Episodes controlled with 2 infusions |
1
|
7
|
8
|
| Bleeding episodes controlled with ≥ 3 infusions |
1
|
2
|
3
|
| Title | Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed |
|---|---|
| Description | Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring. - Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. - Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens. |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants in the Full Analysis Set who had bleeding episodes |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 7 | 7 | 14 |
| Measure Bleeding Episodes | 11 | 15 | 26 |
| Excellent |
9
|
4
|
13
|
| Good |
2
|
10
|
12
|
| Fair |
0
|
1
|
1
|
| None |
0
|
0
|
0
|
| Title | Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR) |
|---|---|
| Description | The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) * 365.25. |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [Bleeding episodes per year] |
1.9
(1.89)
|
3.4
(3.93)
|
2.7
(3.14)
|
| Title | Consumption of BAX326: Number of Infusions Per Month |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [Infusions per month] |
6.8
(0.44)
|
7.2
(0.40)
|
7.0
(0.44)
|
| Title | Consumption of BAX326: Number of Infusions Per Year |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [Infusions per year] |
82.1
(5.27)
|
85.9
(4.79)
|
84.1
(5.27)
|
| Title | Consumption of BAX326: Weight-adjusted Consumption Per Month |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [IU/kg per month] |
393.4
(50.53)
|
414.8
(58.44)
|
404.6
(54.66)
|
| Title | Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized) |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Mean (Standard Deviation) [IU/kg per year] |
4720.9
(606.31)
|
4978.2
(701.26)
|
4855.1
(655.93)
|
| Title | Consumption of BAX326: Weight-adjusted Consumption Per Event |
|---|---|
| Description | Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs). |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Consumption of BAX326 for prophylactic infusions |
56.3
(10.29)
|
56.2
(6.55)
|
56.2
(8.34)
|
| Consumption of BAX326 for infusions to treat BEs |
57.6
(11.87)
|
62.1
(16.00)
|
59.9
(13.74)
|
| Title | Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX) |
|---|---|
| Description | If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data. |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Safety: Number of Participants With Thrombotic Events |
|---|---|
| Description | |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs |
|---|---|
| Description | Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number with CS changes in haematology params |
0
0%
|
2
16.7%
|
2
8.7%
|
| Number with CS changes in clin. chemistry params |
0
0%
|
0
0%
|
0
0%
|
| Number with CS changes in vital signs |
0
0%
|
0
0%
|
0
0%
|
| Title | Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) |
|---|---|
| Description | If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. |
| Time Frame | Throughout study period (approximately 17 months) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Number who developed antibodies to CHO proteins |
0
0%
|
0
0%
|
0
0%
|
| Number who developed antibodies to rFurin |
0
0%
|
0
0%
|
0
0%
|
| Title | Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score |
|---|---|
| Description | For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline. |
| Time Frame | Baseline and 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants in the Full Analysis Set who had PedsQL™ data for baseline and 6 months |
| Arm/Group Title | Full Analysis Set |
|---|---|
| Arm/Group Description | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 16 |
| Peds-QL 2-4 (N=4) |
3.27
(10.119)
|
| Peds-QL 5-7 (N=2) |
-7.07
(6.917)
|
| Peds-QL 8-12 (N=10) |
4.02
(11.038)
|
| Title | Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score |
|---|---|
| Description | The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. |
| Time Frame | Baseline and 6 months |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants in the Full Analysis Set who had Haemo-QoL data for baseline and 6 months |
| Arm/Group Title | Full Analysis Set |
|---|---|
| Arm/Group Description | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 10 |
| Mean (Standard Deviation) [Scores on a scale] |
-0.18
(0.456)
|
| Title | Health Resource Use: Number of Hospitalizations |
|---|---|
| Description | The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. |
| Time Frame | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| Week 5 (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 13 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Week 26 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Title | Health Resource Use: Length of Hospitalization |
|---|---|
| Description | The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. |
| Time Frame | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 1 | 2 | 3 |
| PK assessment |
NA
|
NA
|
NA
|
| Week 5 |
NA
|
NA
|
NA
|
| Week 13 (N=1, 1, 2) |
2.0
|
4.0
|
3.0
|
| Week 26 (N=NA, 1, 1) |
NA
|
13.0
|
13.0
|
| Title | Health Resource Use: Unscheduled Doctor's Office Visits |
|---|---|
| Description | The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. |
| Time Frame | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants who had unscheduled visits to a doctor's office |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| PK assessment (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 5 (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 13 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Week 26 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Title | Health Resource Use: Emergency Room Visits |
|---|---|
| Description | The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. |
| Time Frame | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| PK assessment (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 5 (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 13 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Week 26 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Title | Health Resource Use: Days Lost From School |
|---|---|
| Description | The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. |
| Time Frame | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants who missed days from school |
| Arm/Group Title | Pediatric Participants < 6 Years of Age | Pediatric Participants 6 to < 12 Years of Age | Full Analysis Set |
|---|---|---|---|
| Arm/Group Description | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. | Comprised of all participants who received at least one infusion of study product |
| Measure Participants | 11 | 12 | 23 |
| PK assessment (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 5 (N=11, 12, 23) |
0.0
|
0.0
|
0.0
|
| Week 13 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
| Week 26 (N=11, 11, 22) |
0.0
|
0.0
|
0.0
|
Adverse Events
| Time Frame | Throughout study period (approximately 17 months) | |
|---|---|---|
| Adverse Event Reporting Description | AEs were monitored from the screening visit until the study completion/termination visit. | |
| Arm/Group Title | Full Analysis Set | |
| Arm/Group Description | Comprised of all participants who received at least one infusion of study product | |
All Cause Mortality |
||
| Full Analysis Set | ||
| Affected / at Risk (%) | # Events | |
| Total | / (NaN) | |
Serious Adverse Events |
||
| Full Analysis Set | ||
| Affected / at Risk (%) | # Events | |
| Total | 3/23 (13%) | |
| Infections and infestations | ||
| Device related infection | 1/23 (4.3%) | 1 |
| Injury, poisoning and procedural complications | ||
| Humerus fracture | 1/23 (4.3%) | 1 |
| Musculoskeletal and connective tissue disorders | ||
| Hemarthrosis | 1/23 (4.3%) | 1 |
| Skin and subcutaneous tissue disorders | ||
| Subcutaneous hemorrhage | 1/23 (4.3%) | 1 |
Other (Not Including Serious) Adverse Events |
||
| Full Analysis Set | ||
| Affected / at Risk (%) | # Events | |
| Total | 15/23 (65.2%) | |
| Gastrointestinal disorders | ||
| Abdominal pain | 2/23 (8.7%) | 2 |
| Toothache | 2/23 (8.7%) | 2 |
| Infections and infestations | ||
| Bronchitis | 2/23 (8.7%) | 2 |
| Nasopharyngitis | 2/23 (8.7%) | 3 |
| Respiratory tract infection viral | 2/23 (8.7%) | 2 |
| Rhinitis | 2/23 (8.7%) | 4 |
| Upper respiratory tract infection | 3/23 (13%) | 3 |
| Investigations | ||
| Immunology test abnormal | 6/23 (26.1%) | 8 |
| Nervous system disorders | ||
| Headache | 2/23 (8.7%) | 2 |
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Baxalta's agreements with PIs may vary per individual PI, but contain common elements. For this study, PIs may be restricted from independently publishing results without prior approval.
Results Point of Contact
| Name/Title | Study Director |
|---|---|
| Organization | Shire |
| Phone | +1 866 842 5335 |
| ClinicalTransparency@shire.com |
Responsible Party:
Baxalta now part of Shire
ClinicalTrials.gov Identifier:
NCT01488994
Other Study ID Numbers:
- 251101
- 2011-002437-19
First Posted:
Dec 9, 2011
Last Update Posted:
May 20, 2021
Last Verified:
Apr 1, 2021